Eltrombopag+hATG+CsA vs. hATG+CsA in Children With Severe AA

• Register: ClinicalTrials.gov
• Last refreshed on: 22 March 2021
• Main ID: NCT03413306
• Date of registration: 22/12/2017
• Prospective Registration: No
• Primary sponsor: Federal Research Institute of Pediatric Hematology, Oncology and Immunology
• Public title: Eltrombopag+hATG+CsA vs. hATG+CsA in Children With Severe AA
• Scientific title: A Phase II Multicenter Randomized Study of Eltrombopag Combined With Cyclosporine and hATG Versus hATG and CsA as First Line Treatment in Pediatric Patients With Severe Acquired Aplastic Anemia
• Date of first enrolment: December 10, 2016
• Target sample size: 100
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/show/NCT03413306
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Russian Federation

Contacts

• Name: Olga Goronkova, MD

• Name: Zhanna Shekhovtsova

• Name: Galina Novichkova
• Affiliation: Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology

• Name: Alexei Maschan
• Affiliation: Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Clinical diagnosis of severe and very severe Aplastic anemia

2. 2 - 18 years old

3. Written informed consent signed by a parent or legal guardian prior to initiation of
any study specific procedure.

4. Absence of HLA-identical family member

Exclusion Criteria:

1. myelodysplastic syndrome 4. Prior immunosuppressive therapy 5. Patients with hepatic,
renal or cardiac failure, or any other life- threatening concurrent disease 6.
hypersensitivity to any of the component medications 7. Creatinine >2.5 mg/dL× the upper
limit of normal, 8. Total bilirubin >1.5 × the upper limit of normal mg/dL , 9. Aspartate
aminotransferase (AST) or alanine aminotransferase (ALT) >3-5 × the upper limit of normal



Age minimum: 2 Years
Age maximum: 18 Years
Gender: All

Health Condition(s) or Problem(s) studied

Acquired Aplastic Anemia

Intervention(s)

Drug: Eltrombopag

Drug: IST (ATG + CsA)

Primary Outcome(s)

ORR (CR + PR) [Time Frame: 4 months]

Secondary Outcome(s)

Comparison of cumulative incidence of adverce effects in two arms [Time Frame: 2 years]

Duration of hematologic response [Time Frame: 2 years]

Cumulative incidences of clonal evolution [Time Frame: 2 years]

Neutrophil count [Time Frame: 4 and 6 months]

Cumulative incidence of adverce effects [Time Frame: 4 months]

Cumulative incidence of PNH population occurrence and clinical hemolytic PNH occurrence [Time Frame: 2 years]

Cumulative incidence of response [Time Frame: 4 and 6 months]

Event-free survival [Time Frame: 2 years]

Hemoglobin [Time Frame: 4 and 6 months]

Platelet count [Time Frame: 4 and 6 months]

Cumulative incidence of relapse [Time Frame: 2 years]

Overall survival [Time Frame: 2 years]

Secondary ID(s)

NCPHOI-2016-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors